Review
Medicine, General & Internal
Paula Salmikangas, Bjorn Carlsson, Christophe Klumb, Tatiana Reimer, Steffen Thirstrup
Summary: Potency is a critical quality attribute of biological medicinal products that reflects their biological activity. For timely release and comparability studies, quantitative in vitro assays are necessary. Cell and Gene Therapy Products (CGTs) present challenges in potency testing due to their complexity.
FRONTIERS IN MEDICINE
(2023)
Review
Immunology
Chiara Capelli, Carolina Cuofano, Chiara Pavoni, Simona Frigerio, Daniela Lisini, Sara Nava, Michele Quaroni, Valentina Colombo, Francesco Galli, Svetlana Bezukladova, Paola Panina-Bordignon, Giuseppe Gaipa, Patrizia Comoli, Giulio Cossu, Gianvito Martino, Andrea Biondi, Martino Introna, Josee Golay
Summary: Advanced Therapy Medicinal Products (ATMPs) based on somatic cells expanded in vitro, with or without genetic modification, is a rapidly growing area of drug development. Potency assays are crucial for assessing the quality of ATMPs, and biomarkers may serve as substitutes for complex functional potency tests in predicting the efficacy of these cell-based drugs in vivo.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Chemistry, Analytical
Yu Lei, Zhou Yong, Junzhi Wang
Summary: Potency assays are crucial for the development, registration, and quality control of biological products. Genetically modified (GM) cell line-based potency assays are widely used for the quality control of various biological products. This review provides insights into the principles, technologies, and concerns related to the development and application of GM cell-based potency assays.
JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS
(2023)
Review
Biotechnology & Applied Microbiology
Sha Sha, Andrew J. Maloney, Georgios Katsikis, Tam N. T. Nguyen, Caleb Neufeld, Jacqueline Wolfrum, Paul W. Barone, Stacy L. Springs, Scott R. Manalis, Anthony J. Sinskey, Richard D. Braatz
Summary: The article focuses on analyzing the bottlenecks in rAAV production during cell culture, comparing differences between wild-type and recombinant systems, and proposing future directions for improvement.
BIOTECHNOLOGY ADVANCES
(2021)
Article
Food Science & Technology
Pricila P. Nass, Tatiele C. do Nascimento, Andressa S. Fernandes, Patricia A. Caetano, Veridiana V. de Rosso, Eduardo Jacob-Lopes, Leila Q. Zepka
Summary: This study investigated the effects of different microalgal matrices on the bioaccessibility and uptake of carotenoids and chlorophylls by Caco-2 cells. The results showed that the matrix of liposoluble pigment emulsion had the highest promotion effect on the bioaccessibility and cellular uptake of carotenoids and chlorophylls. Therefore, Chlorella vulgaris and Arthrospira platensis could be considered as ingredients in formulations targeting carotenoids and chlorophylls.
FOOD RESEARCH INTERNATIONAL
(2022)
Article
Microbiology
Arthur H. Totten, Anna Julia Adams, Hyunmi K. Halas, James E. T. Gebo, Amanda D. East, Anna F. Lau
Summary: Testing of cellular therapy products for Mycoplasma is required by the FDA to ensure sterility and safety. Various molecular assays have been marketed for faster testing, but their performance is not well-documented. This study evaluated the performance of five commercial molecular assays and found that not all assays met the LOD required by pharmacopeia standards.
JOURNAL OF CLINICAL MICROBIOLOGY
(2023)
Review
Pharmacology & Pharmacy
Yolanda Diebold, Laura Garcia-Posadas
Summary: The conjunctiva, as a complex ocular tissue, plays a crucial role in protecting the ocular surface, but faces challenges in treatment. Cell therapy and tissue engineering techniques show promise in providing better treatment options for conjunctival diseases.
Article
Cell & Tissue Engineering
Toshimitsu Tanaka, Keiji Yoshimura, Ryan Chang, Bryan Choi, Ying Gai, Pawan Kumar Gupta, Udaykumar Kolkundkar, Shing-Mou Lee, Sunray Lee, Wenbin Liao, Xiang Zhao, Koji Takakura
Summary: This study investigated the guidelines and pharmacopeia for biological ancillary materials (AMs) used in the manufacture of cell and gene therapy (CGT) products in Asian countries and compared them with the guidelines from the United States and European Union. The authors categorized the guidelines into general risk assessment guidelines and specific guidelines for AMs. They also compiled in-depth descriptions of regulations in China, India, Japan, Korea, and Taiwan. The lack of common requirements and limited availability of specific AM guidelines highlight the need for a risk assessment approach considering biological origin, traceability, and control in the CGT manufacturing process. (c) 2022 International Society for Cell & Gene Therapy. Published by Elsevier Inc.
Review
Pharmacology & Pharmacy
Fatima Aerts-Kaya, Niek P. van Til
Summary: Leukodystrophies are a group of genetic degenerative brain disorders that can be lethal if not treated. Gene therapy offers promising results for the treatment of leukodystrophies, and this article discusses the potential interventions of gene therapy in certain types of leukodystrophies, such as X-linked adrenoleukodystrophy and metachromatic leukodystrophy. The advantages and disadvantages of different gene therapy approaches are also discussed, along with the ongoing developments in the field of molecular technologies for treating leukodystrophies in the future.
Review
Biochemistry & Molecular Biology
Georgy Sagaradze, Anna Monakova, Anastasia Efimenko
Summary: Adult stem cells are regulated by their specific microenvironments called stem cell niches, and dysfunction of the niche components can lead to chronic or acute disorders. To address this, regenerative medicine treatments such as gene and cell therapy are being investigated, with a focus on mesenchymal stromal cells (MSCs) and their secretomes. However, the development of MSC secretome-based products is not fully regulated, which has complicated their clinical translation and resulted in failed trials. This review discusses the development of potency assays for MSC secretome-based products, specifically their effects on stem cell niches and spermatogonial stem cell niche.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Review
Endocrinology & Metabolism
Lara E. Graves, David J. Torpy, P. Toby Coates, Ian E. Alexander, Stefan R. Bornstein, Brigette Clarke
Summary: Primary adrenal insufficiency occurs in both adults and children, with autoimmune adrenalitis being the main cause in adults and congenital adrenal hyperplasia (CAH) in children. Current treatment involves oral replacement of cortisol, but there are issues such as poor quality of life and dosing uncertainty. Adrenocortical cell therapy and gene therapy offer potential solutions to these problems, including allogeneic adrenocortical cell transplantation for physiological cortisol secretion and gene therapy to replace or repair defective genes. This review discusses the advances in these therapies for patients with primary adrenal insufficiency.
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
(2023)
Article
Medicine, Research & Experimental
Tereza Buchtova, Lucie Beresova, Katarina Chroma, Tomas Pluhacek, Tibor Beres, Dominika Kaczorova, Petr Tarkowski, Jiri Bartek, Martin Mistrik
Summary: Cannabinoids derived from Cannabis sativa L. have become more accessible for public consumption in the form of cannabis products. Some cannabis-derived drugs have been approved for managing diseases and chemotherapy-induced nausea and vomiting. Preclinical data using human cell culture models suggests that cannabidiol and cannabis extracts may counteract the anticancer effects of platinum-based drugs commonly used in therapy.
BIOMEDICINE & PHARMACOTHERAPY
(2023)
Article
Nanoscience & Nanotechnology
Dagmara J. Trojanowska, Giulia Suarato, Clarissa Braccia, Andrea Armirotti, Fabrizio Fiorentini, Athanassia Athanassiou, Giovanni Perotto
Summary: In this study, keratin particles were produced via isoelectric precipitation and found to contain the amino acid sequence LDV, which can promote cell adhesion and growth orientation. Microcontact printing was used to facilitate the adhesion and growth of dermal fibroblast cells on the keratin particles.
ACS APPLIED NANO MATERIALS
(2022)
Editorial Material
Oncology
Erica Lyons, Charles Line, Jessica J. Lee
Summary: Preventing CINV is a crucial part of cancer treatment plans, and the FDA's draft guidance for industry aims to facilitate the clinical development of drugs for this purpose, expediting the process and providing safe and effective therapies to patients in need. Leveraging guidance documents can help improve communication between regulatory agencies, academic community, patient advocacy groups, and pharmaceutical industry stakeholders.
CLINICAL CANCER RESEARCH
(2021)
Review
Pharmacology & Pharmacy
Marina Lopez-Paniagua, Ana de la Mata, Sara Galindo, Francisco Blazquez, Margarita Calonge, Teresa Nieto-Miguel
Summary: ATMPs are innovative and complex biological products for human use, including cell therapy, tissue engineering, gene therapy, and combined products with medical devices. The development of ATMPs for treating eye diseases has rapidly expanded in recent years, offering novel therapeutic approaches for incurable conditions.
